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Mavorixafor

Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome

Ensayo de X4 Pharmaceuticals, Inc en sindrome WHIM.

Fase

Fase 3

Estado

Completado

Participantes

31

previstos

Centros

23

Fin del objetivo primario

dic 2025

fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, aleatorizado, doble ciego. Comenzó en oct 2019.

Qué mide

Periodo aleatorizado controlado con placebo: tiempo (en horas) por encima del umbral de recuento absoluto de neutrófilos (TAT-ANC en horas) de ≥ 500 células/microlitro (µL) durante un periodo de 24 horas (Time 0 (pre-dose, up to 15 minutes prior), 30, 60, and 90 min (each ± 5 min) and 2, 3, 4, 8, 12, 16, and 24 hours (each ± 15 min) post-dose at Baseline, Weeks 13, 26, 39, and 52) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroThis study has a double-blind, Randomized Placebo-Controlled Period and an Open-Label Period. The primary objective of the Randomized Placebo-Controlled Period is to demonstrate the efficacy of mavorixafor in participants with WHIM syndrome as assessed by increasing levels of circulating neutrophils compared with placebo, and relative to a clinically meaningful threshold. The primary objective of the Open-Label Period is to evaluate the safety and tolerability of mavorixafor in participants with WHIM syndrome. Participants are allowed to continue treatment in the Open-Label Period, if regionally applicable, until mavorixafor becomes commercially available, or until the study is terminated by the Sponsor.

Más ensayos de XFOR

Todos los de XFOR, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT03995108), actualizada en jul 2026.